AAVs in Retinal Gene Therapy
Type
Blog Post
Published
July 31, 2018, 12:55 p.m.
... was in its infancy, but the eye was identified early on as an ideal target. It is an easily accessible...dog, was characterized and found to suffer from early and severe visual impairment that faithfully recapitulated...for ocular conditions. However, AdV was abandoned early on due to its considerable immunogenicity and its...candidate therapies for these diseases.
Riding on the early successes of retinal gene therapy, an ever-growing... to name a few.
CRISPR-based tools are also particularly well suited to be combined with AAV for in vivo...clinical study combining these tools may start as early as next year.
Twenty years after falling from grace...